Study of Lademirsen (SAR339375) in Patients With Alport Syndrome

Stopped early · Phase 2 · Has a placebo group

Conditions studied: Alport Syndrome

In brief

Primary Objectives: * To assess the efficacy of lademirsen (SAR339375) in reducing the decline in renal function. * To assess the safety and tolerability of lademirsen (SAR339375) in participants with Alport syndrome. Secondary Objectives: * To assess plasma pharmacokinetic (PK) parameters of the parent compound and its active major metabolite. * To assess the potential formation of anti-drug antibodies (ADAs) following administration of lademirsen (SAR339375). * To assess the pharmacodynamic effect of lademirsen (SAR339375) on miR-21 and on changes in renal injury and function biomarkers.

Key facts

Study ID

NCT02855268

Run by

Genzyme, a Sanofi Company

People needed

43

Starts

2019-11-02

Expected to finish

2022-09-22

Last updated by the study team

2025-09-11

Who can join

Age: 18 and older, up to 55. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Male or female.
• Confirmed diagnosis of Alport syndrome
• Clinical diagnosis (hematuria, family history, hearing loss, ocular change), AND
• Genetic confirmation of Alport Syndrome in the participant or the family member, OR
• Kidney biopsy showing glomerular basement membrane abnormalities (e.g., significant thinning, thickening, irregularity or lucencies) consistent with Alport Syndrome.
• Age 18-55 years old.
• eGFR > 35 ml/min/1.73m\^2 and <90 mL/min/1.73m\^2 (based on CKD-EPI) at screening.
• Renal Function Criteria (participants must have met at least one of the following CRITERIA A, B or C):
• A) Decline in eGFR of >=4 mL/min/1.73 m\^2/year (eGFR slope <= -4) based on a linear regression slope analysis of >=4 eGFR measurements within 3 years prior to the study and with a minimum of 2-year time span (the last, of the screening measurement, and first eGFR measurements should be separated by at least 2 years). eGFR was calculated by using either the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation.
• B) proteinuria (UPCR) >2000 mg/g (UACR>1000 mg/g).
• C) Age and sex adjusted eGFR (based on CKD-Epi; male 18-23 eGFR<90 mL/min/1.73m\^2
• ACE inhibitor and/or ARB, the dosing regimen should be stable for at least 30 days prior to screening.
• Sexually active female participants of childbearing potential and sexually mature male participants must have agreed to practice true abstinence in line with their preferred and usual lifestyle or to use two acceptable effective methods of contraception for the entire duration of the study and for at least 6 weeks after last dose.
• Negative drug screen for opiates, cocaine, heroin, phencyclidine, amphetamines (including ecstasy), barbiturates, benzodiazepines, and cannabinoids. At the Investigator's discretion, participants prescribed benzodiazepines, cannabinoids, or opiates with positive results on a drug screen were allowed.
• Negative screening results for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, and human immunodeficiency virus (HIV) antibody.
• Normal biological tests.
• Able to understand all study procedures in the informed consent form (ICF) and to comply with all aspects of the protocol.

You may not qualify if…

• Causes of chronic kidney disease aside from Alport syndrome (including but not limited to other heritable disorders leading to chronic kidney disease, diabetic nephropathy, hypertensive nephropathy, lupus nephritis, IgA nephropathy).
• End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transplantation.
• Any clinically significant illness within 30 days before screening or surgical or medical condition (other than Alport syndrome) that could interfere with the participant's study compliance; confound the study results; impact participant safety; or significantly alter the absorption, distribution, metabolism, or excretion of drugs.
• Weight > 110 kg.
• Any history of active malignancy within the last 1 year (history of localized basal cell or squamous cell carcinoma and cervical carcinoma in situ that has been excised/appropriately treated or a fully excised malignant lesion with a low probability of recurrence will not be considered exclusionary).
• Prior treatment with Bardoxolone within 90 days prior to screening.
• History or presence of alcoholism or drug abuse within 2 years before screening or other concurrent social conditions that would potentially interfere with the participant's study compliance, at the discretion of the Investigator.
• Participation in a recent investigational study and receipt of an investigational drug or investigational use of a licensed drug within 30 days or 5 half-lives, whichever was longer, prior to screening.
• History or presence of hypersensitivity or idiosyncratic, allergic, or other clinically significant reaction to the study drug (including placebo), inactive ingredients, or related compounds (e.g., other oligonucleotide products).
• Any other condition or circumstance that, in the opinion of the Investigator, may make the participant unlikely to complete the study or comply with study procedures and requirements, or may pose a risk to the participant's safety and well-being.
• The above information was not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.

Where it is running

• Investigational Site Number :8400002 — Los Angeles, California, United States
• University of Minnesota Childrens' Hospital_Investigational Site Number :8400003 — Minneapolis, Minnesota, United States
• Columbia University Medical Center_Investigational Site Number :8400004 — New York, New York, United States
• The Cleveland Clinic Foundation_Investigational Site Number :8400001 — Cleveland, Ohio, United States
• University of Utah_Investigational Site Number :8400005 — Salt Lake City, Utah, United States
• Investigational Site Number :0360003 — Herston, Queensland, Australia
• Investigational Site Number :0360001 — Parkville, Victoria, Australia
• Investigational Site Number :0360002 — Nedlands, Western Australia, Australia
• Investigational Site Number :1560001 — Beijing, China
• Investigational Site Number :1560002 — Beijing, China
• Investigational Site Number :1560004 — Guangzhou, China
• Investigational Site Number :2500001 — Paris, France
• Investigational Site Number :2500002 — Toulouse, France
• Uniklinik Köln, Innere Medizin II - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin_Investigational Site Number :2760001 — Cologne, Germany
• Universitätsmedizin Göttingen, Klinik für Nephrologie und Rheumatologie_Investigational Site Number :2760002 — Göttingen, Germany
• Investigational Site Number :7240005 — Córdoba, Andalusia, Spain
• Investigational Site Number :7240001 — Barcelona, Barcelona [Barcelona], Spain
• Investigational Site Number :7240004 — Barcelona, Barcelona [Barcelona], Spain
• Investigational Site Number :7240002 — Madrid / Madrid, Madrid, Comunidad de, Spain
• Investigational Site Number :7240003 — Granada, Spain
• Investigational Site Number :8260001 — London, London, City of, United Kingdom
• Investigational Site Number :8260002 — Nottingham, Nottinghamshire, United Kingdom
• Investigational Site Number :8260003 — Newcastle upon Tyne, United Kingdom

Full record on ClinicalTrials.gov

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